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Vertex Announces Presentation of New Data on VX-770 and VX-809 at North American Cystic Fibrosis Conference
Complete 48-week data from the Phase 3 ENVISION study will be presented for the first time. ENVISION evaluated VX-770 in children ages 6 to 11 years with at least one copy of the G551D mutation in the CFTR gene. Also being presented for the first time are data from a subset of patients in the open-label PERSIST extension study. This rollover study enrolled patients who had completed 48 weeks of treatment (placebo or VX-770) in one of the VX-770 Phase 3 trials and who met certain additional criteria. Data from the first 12 weeks of the rollover study will be presented at the meeting.
In addition, complete data from the first part of a Phase 2 study evaluating multiple combinations of VX-770 and VX-809 in people with two copies of the most common mutation in the CFTR gene, F508del, will be presented for the first time.
"Emerging data from these studies further support the idea that targeting the underlying cause of CF could potentially improve outcomes for people with cystic fibrosis," said
The accepted abstracts are now available in Pediatric Pulmonology: Special Issue: The 25th Annual
VX-770 Abstracts (Oral presentations will also be presented as posters.)
Study 102 (STRIVE): "Efficacy and Safety of VX-770 in Subjects with Cystic Fibrosis and the G551D-CFTR Mutation."
November 5, 2011, 3:35 p.m. PDT(oral). Poster #211.
- Study 103 (ENVISION): "VX-770 in Subjects 6 to 11 Years with Cystic Fibrosis and the G551D-CFTR Mutation." Poster #203.
- Study 104 (DISCOVER): "VX-770 in Subjects with CF and Homozygous for the F508DEL-CFTR Mutation." Poster #206.
- Study 105 (PERSIST): "Long-Term Safety and Efficacy of Investigational CFTR Potentiator, VX-770, in Subjects with CF." Poster #204.
Study 106: "Lung Clearance Index as an Outcome Measure in Cystic Fibrosis Clinical Trials."
November 3, 2011, 11:20 a.m. PDT(oral). Poster #201.
- Study 107: "Hyperpolarized Helium-3 Magnetic Resonance Imaging of CFTR Potentiator Therapy in Subjects with Cystic Fibrosis and the G551D Mutation." Poster #205.
"VX-770 Potentiation of CFTR Forms with Channel Gating Defects In Vitro."
November 4, 2011, 3:20 p.m. PDT(oral). Poster #10.
- "Variability of Nasal Potential Difference Measurements in Clinical Testing of CFTR Modulators." Poster #202.
"VX-809, an Investigational CFTR Corrector, in Combination with VX-770, an Investigational CFTR Potentiator, in Subjects with CF and Homozygous for the F508DEL-CFTR Mutation."
November 5, 2011, 3:50 p.m. PDT(oral). Poster #212.
VX-770 is Vertex's lead medicine in development for the treatment of people with cystic fibrosis. VX-770, known as a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, aims to help the protein function more normally once it reaches the cell surface. In October, Vertex plans to submit both its VX-770 New Drug Application (NDA) in
VX-809 is an investigational oral cystic fibrosis transmembrane conductance regulator (CFTR) modulator for the treatment of cystic fibrosis. VX-809, known as a CFTR corrector, is designed to help the protein reach the cell surface. Vertex is also conducting a Phase 2 clinical trial to evaluate combination regimens of VX-770 and VX-809 in people with two copies of the most common mutation in the CFTR gene, F508del.
About Cystic Fibrosis
Cystic fibrosis is a life-threatening genetic disease affecting approximately 30,000 people in
Collaborative History with
Vertex initiated its CF research program in 1998 as a part of a collaboration with CFFT, the non-profit drug discovery and development affiliate of the
Vertex creates new possibilities in medicine. Our team discovers, develops and commercializes innovative therapies so people with serious diseases can lead better lives.
Vertex scientists and our collaborators are working on new medicines to cure or significantly advance the treatment of hepatitis C, cystic fibrosis, epilepsy and other life-threatening diseases.
Founded more than 20 years ago in
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including statements regarding (i) the aim of Vertex's CF research program; (ii) emerging data from the studies referred to in this press release further supporting the idea that targeting the underlying cause of CF could potentially improve outcomes for people with CF; (iii) the progress Vertex is continuing to make in developing potential new treatments for CF that target the underlying cause of the disease; (iv) Vertex's goal of improving the lives of people with CF and (v) Vertex's plan to submit an NDA and MAA for VX-770 in
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